Sebelipase alfa

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Sebelipase alfa
Clinical data
Trade namesKanuma
AHFS/Drugs.comMonograph
License data
Pregnancy
category
Intravenous infusion
ATC code
Legal status
Legal status
Pharmacokinetic data
Elimination half-life0.1 hours
Identifiers
DrugBank
ChemSpider
  • none
KEGG

Sebelipase alfa, sold under the brand name Kanuma, is a

lysosomal acid lipase (LAL) that is used as a medication for the treatment of lysosomal acid lipase deficiency (LAL-D).[6][7] It is administered via intraveneous infusion.[5] It was approved for medical use in the European Union and in the United States in 2015.[8][6][5][7]

Medical uses

Sebelipase alfa is indicated for long-term enzyme replacement therapy (ERT) in people of all ages with lysosomal acid lipase (LAL) deficiency.[6]

History

Sebelipase was developed by

FDA announced that its approval came from two centers: The Center for Drug Evaluation and Research (CDER) approved the human therapeutic application of the medication, while the Center for Veterinary Medicine (CVM) approved the application for a recombinant DNA construct in genetically engineered chicken to produce rhLAL in their egg whites.[10] At the time it gained FDA approval Kanuma was the first only drug manufactured in chicken eggs and intended for use in humans.[9]

Sebelipase alfa is an orphan drug; its effectiveness was published after a phase 3 trial in 2015.[11] The disease of LAL affects < 0.2 in 10,000 people in the EU.[8]

References

  1. ^ "Sebelipase alfa (Kanuma) Use During Pregnancy". Drugs.com. 14 June 2019. Retrieved 4 May 2020.
  2. ^ "Prescription medicines: registration of new chemical entities in Australia, 2017". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 9 April 2023.
  3. ^ "Prescription medicines and biologicals: TGA annual summary 2017". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 31 March 2024.
  4. ^ "Genetic disorders". Health Canada. 9 May 2018. Retrieved 13 April 2024.
  5. ^ a b c "Kanuma- sebelipase alfa injection, solution, concentrate". DailyMed. 18 December 2018. Retrieved 4 May 2020.
  6. ^ a b c d "Kanuma EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 4 May 2020. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  7. ^ a b "Kanuma (Sebelipase alfa)". U.S. Food and Drug Administration (FDA). 6 January 2016. Retrieved 4 May 2020.
  8. ^ a b "New Drugs Online Report for sebelipase alfa". UK Medicines Information. Archived from the original on 4 March 2016. Retrieved 10 December 2015.
  9. ^
    S2CID 7593902
    .
  10. ^ "FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients". FDA. 8 December 2015. Retrieved 10 December 2015.
  11. PMID 26352813
    .
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